ONCODESIGN (Paris:ALONC) (FR0011766229 - ALONC), a biotechnology
company serving the pharmaceutical industry in the discovery of new
therapeutic molecules to fight cancer and other serious illnesses with
no known effective treatment, will present today with Ipsen (Euronext:
IPN, ADR: IPSEY), a global specialty-driven pharmaceutical company, the
discovery and characterization of ODS2005294, a novel LRRK2 inhibitor
they jointly discovered in a research collaboration started in January
2012. The molecule will be presented at the 45th annual
meeting of the Society for Neurosciences that is taking place October
17-21 in Chicago, IL; this leading global meeting for presentation of
novel discoveries in neuroscience is attended by over 30,000 experts in
the field from over 80 countries.

ODS2005294 is a potent and selective small molecule derived from
Oncodesign’s Nanocyclix® technology. It shows good in vivo
oral exposure and reaches high levels in the brain, where it
demonstrates dose-related inhibition of LRRK2 phosphorylation with no
obvious adverse effect at pharmacological effective doses. ODS2005294 is
the most fully characterized lead of a series of Nanocyclix®
inhibitors that have the potential to address the relevance and the
application of LRRK2 kinase inhibition in the human brain as a potential
treatment for Parkinson’s disease. More advanced molecules in the series
are currently under investigation.

Specific mutations in LRRK2 were identified in 2004 in familial cases of
Parkinson’s disease. Since then, this kinase is considered as one of the
highest potential molecular targets against this neurodegenerative
disorder. Despite being the subject of massive research efforts since
2005, no inhibitors have yet entered clinical stages of development,
because of the difficulty to find potent, selective inhibitors that
readily cross the Blood-Brain Barrier and that are completely safe for
use in a chronic disease setting.

At the same meeting, in a French multi-center approach, clinical
scientists from the Brain and Spine Institute (ICM, Paris) and the
French clinical research network for Parkinson’s disease and Movement
Disorders (NS-Park, FCRIN), showed strong inhibition of LRRK2 induced
phosphorylation on human peripheral blood mononuclear cells (hPBMCs)
from Parkinson patients by ODS2005294.

“This new exciting LRRK2 inhibitor shows the progress we have made in
the collaborative program with Ipsen since 2012. The combination of
Oncodesign’s Nanocyclix® technology for next
generation kinase inhibitors with Ipsen’s recognized expertise in CNS
research has allowed us to identify a class of molecules that has the
potential to advance towards candidate selection. Both Oncodesign and
Ipsen are committed to continue their efforts to improve the lives of
Parkinson’s patients”, said Philippe Genne, Ph.D., Chief
Executive Officer and founder of Oncodesign.

“ODS2005294 is a potent and selective orally active molecule that
reaches high levels in the brain and that has been shown to inhibit the
phosphorylation of LRRK2 in the brain in animal models without side
effects at efficacious doses. The work by clinical scientists on human
blood cells shows that our compound also potently blocks the kinase
activity of the human form of LRRK2, an essential condition to further
advance the program", added Jan Hoflack, Ph.D., Chief
Scientific Officer of Oncodesign.

About Parkinson’s disease

Parkinson’s disease (PD) is the most common neurodegenerative movement
disorder afflicting 1% of the population aged 65 years and older.
Clinical features include bradykinesia, rigidity and tremor. PD is
characterized by progressive loss of dopaminergic neurons and
accumulation of aggregation of α–synuclein protein in the brain. Only
dopamine replacement therapy which compensates the dopamine neuronal
loss reduces with some efficacy motor symptoms in PD patients but does
not stop or slow the neurodegenerative process. At present, there are no
proven neuroprotective or neurorestorative therapies. Disease
modification is thus the most important goal in PD.

About LRRK2 target

Although PD is regarded as a sporadic disorder, 5-10% of PD cases are
genetically inherited as familial. LRRK2 mutations represent the highest
risk of familial PD and are also observed in sporadic patients.
Pathological characteristics and clinical symptoms observed in patients
carrying LRRK2 mutations are indistinguishable between familial and
sporadic patients. LRRK2 is a multidomain protein which contains both
GTPase and Kinase enzymatic activities where most pathogenic mutations
are located. LRRK2 inhibition represents a potential neuroprotective
therapeutic target for the treatment of PD.

Founded 20 years ago by Dr. Philippe Genne, the Company’s CEO and
Chairman, ONCODESIGN is a biotechnology company that maximizes the
pharmaceutical industry’s chances of success in discovering new
therapeutic molecules to fight cancer and other serious illnesses with
no known efficient treatment. Backed by unique experience acquired
through more than 600 clients, including the world’s largest
pharmaceutical companies, and relying on a comprehensive technological
platform combining state-of-the-art medicinal chemistry, advanced animal
modeling and medical imaging, ONCODESIGN is able to predict and identify
for every molecule, very upstream, its therapeutic use and its potential
to become an efficient drug. Applied to kinase inhibitors, molecules
that represent a market estimated at over 40 billion dollars in 2016 and
accounting for almost 25% of the pharmaceutical industry’s R&D
investments, ONCODESIGN’s technology has already enabled the targeting
of 7 promising molecules with substantial therapeutic potential, in
oncology and elsewhere, and the signing of partnerships, potentially
worth €350 million in upfront payments should predefined milestones be
reached, with pharmaceutical groups Sanofi, Ipsen and UCB. Based in
Dijon, France, in the heart of the town’s university and hospital hub,
ONCODESIGN has 95 staff.